Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor.

• Register: EUCTR
• Last refreshed on: 3 August 2020
• Main ID: EUCTR2016-002025-11-DE
• Date of registration: 07/10/2016
• Prospective Registration: Yes
• Primary sponsor: Alexion Pharmaceuticals Incorporated
• Public title: Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor.
• Scientific title: A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Date of first enrolment: 20/12/2016
• Target sample size: 214
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-002025-11
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: yes; Open: yes; Single blind: no; Double blind: no; Parallel group: yes; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: yes; Placebo: no; Other: no; Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Argentina, Australia, Austria, Belgium, Brazil, Canada, Colombia, Czech Republic, Denmark, Estonia, Finland, France, Germany, Italy, Japan, Korea, Republic of, Malaysia, Mexico, Netherlands, Poland, Portugal, Russian Federation, Singapore, Spain, Sweden, Taiwan, Thailand, Turkey, United Kingdom, United States

Contacts

• Name: European Clinical Trial Information
• Affiliation: Alexion Europe SAS

• Name: European Clinical Trial Information
• Affiliation: Alexion Europe SAS

Key inclusion & exclusion criteria

Inclusion criteria:
1. Male or female = 18 years of age
2. PNH diagnosis confirmed by documented by high-sensitivity flow
cytometry
3. Presence of 1 or more of the following PNH-related signs or symptoms within 3 months of Screening: fatigue, hemoglobinuria, abdominal pain,
shortness of breath (dyspnea), anemia (hemoglobin <10 g/dL), history of a major adverse vascular event (including thrombosis), dysphagia, or
erectile dysfunction; or history of pRBC transfusion due to PNH.
4. LDH level = 1.5 × ULN at screening.
5. Documented meningococcal vaccination not more than 3 years prior to, or at the time of, initiating study treatment
6. Female patients of childbearing potential must use highly effective
contraception starting at screening and continuing until at least 8 months after the last dose of ALXN1210
7. Willing and able to give written informed consent and comply with study visit schedule

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 192
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 22

Exclusion criteria:
1. Treatment with a complement inhibitor at any time
2. History of bone marrow transplantation
3. Body weight < 40 kilograms
4. Females who are pregnant, breastfeeding or who have a positive pregnancy test at screening or Day 1
5. Participation in another interventional clinical study or use of any experimental therapy within 30 days before initiation of study drug on
Day 1 in this study or within 5 half-lives of that investigational product, whichever is greater.
6. History of or ongoing major cardiac, pulmonary, renal, endocrine, or hepatic disease that, in the opinion of the investigator or sponsor, would
preclude participation
7. Unstable medical conditions (eg, myocardial ischemia, active gastrointestinal bleed, severe congestive heart failure, anticipated need
for major surgery within 6 months of randomization, coexisting chronic anemia unrelated to PNH)


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857

Intervention(s)

Product Code: ALXN1210
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Current Sponsor code: ALXN1210
Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-

Trade Name: Soliris
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: ECULIZUMAB
CAS Number: 219685-50-4
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-

Primary Outcome(s)

Primary end point(s): - Percentage of patients who achieve transfusion avoidance (TA)
- Normalization of lactate dehydrogenase (LDH) levels

Timepoint(s) of evaluation of this end point: 26 weeks

Secondary Objective: - safety and tolerability of ALXN1210
- efficacy
- PK/PD and immunogenicity
- long-term safety and efficacy
- evaluate the safety and efficacy in patients who switch from eculizumab to ALXN1210 in the Extension Period
- quantify identified specific safety concerns during treatment with ALXN1210, including meningococcal infections, serious hemolysis after drug discontinuation in PNH, immunogenicity, serious infections, malignancies and hematologic abnormalities, and during pregnancy and breastfeeding.

Main Objective: The purpose of this study is to assess ALXN1210 compared to eculizumab in adult patients with PNH who have never been treated with a complement inhibitor.

Secondary Outcome(s)

Secondary end point(s): - Percentage change from baseline in lactate dehydrogenase ( LDH) levels
- Change from baseline in quality of life as assessed by the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue
- Percentage of patients with breakthrough hemolysis
- Percentage of patients with stabilized hemoglobin

Timepoint(s) of evaluation of this end point: 26 weeks

Secondary ID(s)

NCT02946463

2016-002025-11-GB

ALXN1210-PNH-301

Source(s) of Monetary Support

Alexion Pharmaceuticals Incorporated

Ethics review

Status: Approved
Approval date: 20/12/2016
Contact:

Results

• Results available: Yes
• Date Posted: 08/07/2020
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2016-002025-11/results